First Drug from Genetically Engineered Animals Approved

The drug is derived from the milk produced by its herd of 200 genetically engineered goats. As the International Herald Tribune explains:

To make its protein, GTC took the human gene for antithrombin and linked it to goat DNA that normally controls production of a protein found in milk. That insured that the antithrombin would be produced only in the milk.

The gene was injected into a one-celled goat embryo, which was then implanted into the womb of a surrogate mother. If it were doing it today, GTC would implant the gene into a skin cell of a desired animal and then produce a clone, a more reliable technique. But Dolly the sheep, the first cloned mammal, had not been created when GTC started its work in the early 1990s.

Once GTC had produced a goat with the human gene that produced the antithrombin in its milk, that "founder" animal was mated with others through conventional breeding to start producing a herd.

Why use goats and other animals to make therapeutic proteins?

Many of the newer protein-based drugs, such as the cancer drugs Avastin and Erbitux and the arthritis drugs Enbrel and Humira, are produced in genetically engineered Chinese hamster ovary cells that are grown in big stainless-steel vats.

But a cell culture factory can cost hundreds of millions of dollars to build. Using livestock eliminates all that steel and shrinks the investment to tens of millions of dollars, said Geoffrey Cox, chief executive of GTC.

One drawback is that given the generation and gestation times of animals, it can take 18 months to start getting protein production from a goat, compared with a few months for cell culture.

Of course, not everyone is happy that goats are exploited in this manner:

"It is a mechanistic use of animals that seems to perpetuate the notion of their being merely tools for human use rather than sentient creatures," the Humane Society of the United States says in its position paper on the practice.

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How does this affect their IP rights? I assume this is a more secure IP model as the process requires access to a unique resource.

I understand for example that the generic availability of certain cancer drugs derived from genetically modified bacteria is imperiled by the argument that current laws do not apply to the products of genetically modified organisms.

This doesn’t seem to be an IP issue.
Someone could breed their own goats to get the protein, but I don’t see this being special in any unique way when compared to manufacturing a drug using more conventional methods.

And if someone stole one of their goats, you have a straight up theft charge, not IP.

I mean, don’t current IP laws cover a drug derived from natural ingredients?

You take natural resource X and use your process to get product Y…patent covers product Y for Z years. This doesn’t seem any different. You take your natural resource X=goats, apply your process Y=genetically engineer them to produce protein that is then used in process to make drug.

Many of the newer protein-based drugs, such as the cancer drugs Avastin and Erbitux and the arthritis drugs Enbrel and Humira, are produced in genetically engineered Chinese hamster ovary cells that are grown in big stainless-steel vats.

But a cell culture factory can cost hundreds of millions of dollars to build. Using livestock eliminates all that steel and shrinks the investment to tens of millions of dollars, said Geoffrey Cox, chief executive of GTC.

Plus you can eat them when they are no longer productive milk producers.

I am also a fan of goat. Sort of a leaner lamb. My brother and I spit-roasted a whole goat 2 summers ago for our annual bbq, and served it with a yellow curry sauce. One of the best things we’ve ever cheffed up.

Oh yeah, this looks like a great idea. And then someone genetically modifies a bull to produce teeth whitener, but instead it grows to 20 feet tall and chases you through a labyrinth with a giant axe. Then you’re all going to look like assholes.

When biologics are approved the production method is fixed circa phase III trials and is a major part of the approval process. In order to get approval to market the drug produced from a goat another party would have to go through the clinical trial process again. Presumedly the molecule as produced by these beasts would be recognised as patent worthy and would be protected this way too.

In Nature Biotechnology this month biotech VC’s are sh*tting their pants over the prospect of capital gains tax being raised to 20% from 15%. Is this a realistic prospect? (The tax hike, not the pants sh*tting.)

I can confirm the expense of cell culture factory based medications. Humira costs ~$2000-$3000 per injection and some people take 1 a week. One would assume the cost would be greatly reduced, especially if clinical trials could begin before the entire herd has been bred.

I grew up about 2 miles from the “high-security farm in central Massachusetts”, the place was a dumpy little crap farm until these folks bought it, and they turned it into quite an awesome little facility. Neither me nor my parents who still live nearby are concerned about any EEEVIL biotech stuff getting in our water or air or food supply or whatever. This is a great thing for mankind, and it was an excellent boost to the two towns that this farm straddles. If anyone is interested, the facility is here:

In addition, one goat will produce about 12 kgs of anti-TNF blocker in 2 weeks, whereas you’d need 10 1000 L bioreactors running to produce that in the same time period (with continious perfusion, anyway). And each bioreactor costs about 1.2 mil to startup, and only lasts 35-60 days…you’d see a significant price drop on new bio drugs within about 10 years.

Unless we went to socialized medicine, which would basically destroy R&D.